Development of a novel observer reported outcome tool as the primary efficacy outcome measure for a rare disease randomized controlled trial

Publication date: Available online 10 November 2017Source: MitochondrionAuthor(s): Peter W. Stacpoole, Jonathan Shuster, John L.P. (Seamus) Thompson, Robert A. Prather, Lee Ann Lawson, Baiming Zou, Richard Buchsbaum, Sara Jo NixonAbstractWe developed an Observer-Reported Outcome (ObsRO) survey instrument to be applied in a multicenter, placebo-controlled, crossover randomized controlled trial of dichloroacetate in children with pyruvate dehydrogenase complex deficiency. The instrument quantifies a subject's at-home level of functionality, as reported by a parent/caregiver, who were instrumental in providing the clinical descriptors and domains that formed the instrument's content. Feasibility testing of the ObsRO tool showed it to be easy to use and comprehensive in capturing the major clinical functional limitations of affected children and requires less than 5 min for a parent/caregiver to complete daily.
Source: Mitochondrion - Category: Biochemistry Source Type: research